Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — 41st Annual J.P. Morgan Healthcare Conference (January 2023)
34 slides from Biomarin Pharmaceutical's conference presentation, Jan 9, 2023
Slide 1: 41st Annual J.P. Morgan Healthcare Conference
41st Annual J.P. Morgan Healthcare Conference January 9, 2023 Transforming Lives Through Genetic Discovery J.J. Bienaimé Chairman and Chief Executive Officer
Slide 2: Forward-looking statements
This non-confidential presentation contains “forward-looking statements” about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the expected financial performance of BioMarin, including, without limitation, preliminary unaudited tota
Slide 3: Scientific Breakthroughs are Foundational to Our Performance
Scientific Breakthroughs are Foundational to Our Performance
Slide 4: Solid, Profitable, "IRA-proof" & Growing Base Business
Solid, Profitable, End to End “IRA-proof” & Growing Base Business Business Partnership Transform ▪ Anchoredpartnership to business by durable mindsettherapies enzyme-based with producing nearly $2B of revenue finance acumen and positive cash flows. Steady revenue growth across the portfolio ▪ High b
Slide 5: Q3 YTD 2022 revenue, operating margin and net income growth
$ Millions Reported Revenue Revenue Growth Operating Margin GAAP Net Income1 Growth Excluding Kuvan® Expansion1 $1,559 11.2% $1,396 $1,385 $142 $1,179 +12% +17% +12.3 ppts +$148M (1.2%) ($6) Q3 YTD 2021 Q3 YTD 2022 Q3 YTD 2021 Q3 YTD 2022 Q3 YTD 2021 Q3 YTD 2022 Q3 YTD 2021 Q3 YTD 2022 1 Operating M
Slide 6: VOXZOGO & ROCTAVIAN $4B-$5B mid-decade revenue
VOXZOGO & ROCTAVIAN >2x $4B - $5B New Base Products $4B-$5B Products ~50% Mid-Decade ~50% $1.85B $2.09B 2021 20221 Mid- End of Decade Decade All other products Anticipated Revenue Growth Enablers Continued growth within Base indication launch of Regulatory approval of U.S. Expansion of VOXZOGO & bas
Slide 7: Sustainable Growth & Profitability
Maximize Base Aggressive Life Indication Revenue Cycle Management Sustainable Growth & Profitability Accelerate Clinical Balance Profits with Pipeline Reinvestments 7
Slide 8: Core Products revenue, +14% CAGR
$ Millions CAGR1 +14% CAGR1 Product 2021 $1,492 2018-21 $1,347 VIMIZIM® $623 +9% $1,175 $1,015 NAGLAZYME® $380 +3% PALYNZIQ® $237 +169% BRINEURA® $128 +48% ALDURAZYME® $123 (3%) Core Products $1,492 +14% 2018 2019 2020 2021 2022E (excluding Kuvan) Core Product Revenue (excluding Kuvan) 8 1 Compounde
Slide 9: VOXZOGO Achondroplasia Market Opportunity and Launch
1,000+ Additional Children Eligible for Treatment upon Anticipated U.S. and E.U. Age Expansions VOXZOGO Achondroplasia Market Opportunity Launch Excellence 2022 Commercial Execution $3B+ • 1 year anniversary Annual 800 18,000+ Market • Over 1,000 children treated at ~17,000 300 Opportunity year-end
Slide 10: ROCTAVIAN Hemophilia A Market Opportunity and Launch Markets
Strategic Market Hemophilia A Base Indicated1 Launch Market Market Access Goal Germany 1Q 2023 $14B+ ~7,000 ~13,000 Cumulative Market Italy 2H 2023 Opportunity France 2H 2023 ~3,000 U.S. 2023 ~3,000 Japan Mid-Decade EMA Indicated U.S. Indicated ROW Indicated Hem A Indicated Hem A Indicated Patients
Slide 11: ROCTAVIAN Clinical Trial Patient Quote
“The impact of gene therapy on my day to day, it has been a life change, it has been a big change...The peace of mind of living day to day, without being afraid of everything. That's the 3 years that I've had so far. A normal life.” “No bleeding, not even once.” ROCTAVIAN Clinical Trial Patient
Slide 12: Outcomes Based Agreements and 2023 Outlook
Outcomes Based Agreements 2023 (OBAs) Outlook • First OBA concluded recently representing • First German patients expected to be multiple German health insurers treated in the coming weeks • Additional completed OBAs in Germany • Comfortable with ROCTAVIAN full-year anticipated in the coming weeks 2
Slide 13: HCP adoption of ROCTAVIAN
~80% of HCPs likely to adopt ROCTAVIAN within 1 year of availability ROCTAVIAN could capture ~35% of severe adult hemophilia A patients, per EU HCPs ~75% of HCPs likely to adopt ROCTAVIAN within 1 year of availability ROCTAVIAN could capture ~40% of severe adult hemophilia A patients, per US HCPs So
Slide 14: U.S. Market Access Plans and Regulatory Progress
U.S. Market Access Plans Advancing: • ICER final assessment confirmed assumed value of ROCTAVIAN at $2.5M • Approached by multiple significant U.S. insurers indicating intent to implement a favorable policy for ROCTAVIAN reimbursement • U.S. outcomes-based agreements will be offered in the form of a
Slide 15: Sustainable Growth & Profitability
Maximize Base Aggressive Life Indication Revenue Cycle Management Sustainable Growth & Profitability Accelerate Clinical Balance Profits with Pipeline Reinvestments 15
Slide 16: VOXZOGO / CNP-Mechanism: Other Genetic Short Stature Conditions
VOXZOGO / CNP-Mechanism: Other Genetic Short Stature (GSS) Conditions 600,000+ GSS conditions currently in the clinic: • Hypochondroplasia • CNP Deficiency • Heterozygous NPR2 mutation • Rasopathy 18,000+ • SHOX Achondroplasia Other GSS • Aggrecan Deficiency Patients Patients (Ages 4-17 years and mo
Slide 17: ROCTAVIAN: Hemophilia A
ROCTAVIAN: Hemophilia A +$3B $32B +$9B $29B +$2B $20B +$4B $18B $14B Initial Indicated Prior Inhibitors Active Inhibitors AAV + Age 15-17 (In the clinic) (In the clinic) (In the clinic) 17 1 ROCTAVIAN market represents a one-time treatment per patient and not annual re-occurring opportunity
Slide 18: Wholly Owned Manufacturing Capabilities
Wholly Owned Manufacturing Capabilities Support Rapid Drug Development and Commercialization Gene Therapy Facilities Biologic Facilities • 2017: Novato, California facility commissioned (ISPE 2018 • 1999: Novato, California facility opened • 2011: Shanbally, Ireland facility acquired Facility of the
Slide 19: Sustainable Growth & Profitability
Maximize Base Aggressive Life Indication Revenue Cycle Management Sustainable Growth & Profitability Accelerate Clinical Balance Profits with Pipeline Reinvestments 19
Slide 20: ROCTAVIAN Phase 3 3-Year and 4-Year Results
Phase 3 6e13 vg/kg dose In Year 3 In Year 4 N=132/112* N=17** Mean 18.8 15.2 FVIII Activity ROCTAVIAN Median 8.4 7.4 PHASE 3 Annualized Mean 1.0 0.8 3-YEAR and 4-Year Bleeding Rate*** (bleeding episodes Median 0.0 0.0 RESULTS per year) Annualized FVIII Mean 8.4 11.1 Utilization (infusions per year)
Slide 21: AAV payload expression in human cells
AAV payload expression in human cells • Non-clinical and clinical studies of AAV episomes indicates that transgene expression is restricted by a AAV payload transcripts/ng RNA transcriptional mechanism • Screen of approved small molecule drugs modulating this mechanism identified several promising c
Slide 22: Research and Early Development
IND- Therapeutic Focus Product Candidate Research Enabling Phase 1 Phase 2 BMN 255 Hyperoxaluria (Small Molecule) BMN 351 DMD (Exon 51 Oligonucleotide) Research and Early Development Musculoskeletal/ BMN 349 A1ATD (Small Molecule) Metabolic Long Acting CNP (Peptide) MSK (Oligonucleotide) 3 Candidate
Slide 23: AGXT Deficiency in PH1
AGXT Deficiency Increased Oxalate Recurrent Kidney Stones In PH1 Inherited Reduced AGXT function Oxalate crystals lead to mutations disrupt the causes increased recurrent kidney stones AGXT gene oxalate and kidney damage 23
Slide 24: BMN 255 Phase 1/2 Single Dose Data
Multi-ascending dose (MAD) Phase 1/2 underway Single BMN 255 Dose in HHVs BMN 255 PH1 & Liver -O- BMN 255 (n=6) Disease - - Mean of BMN 255 -∆- Placebo (n = 2) GO AGT Glycolate Glyoxylate Glycine ---FDA approved Oxalate product for PH1 24
Slide 25: HAE and BMN 331 rationale
SERPING1 haploinsufficiency Reduced C1-Inhibitor Episodes of Severe Angioedema • Available therapies for HAE cannot provide enduring compensation for loss of C1-inhibitor activity • “Troughs” of activity or missed treatment can leave individuals with HAE exposed to severe attacks • Pre-clinical stud
Slide 26: C1-INH antigen levels by visit
First 6 E13 dosing (normal range:19-37 mg/dl) LLN 6e13vg/kg C1-INH antigen (mg/ dl) 2e13vg/kg 2e13vg/kg 26 C1-INH antigen levels (mg/dL) by visit
Slide 27: Product Candidate Key Attributes
Therapeutic Focus Product Candidate / Key Attributes BMN 351 DMD (Exon 51 Oligonucleotide) • > 10x fold enhanced potency by targeting novel splice enhancer • At anticipated muscle concentrations > 20% dystrophin predicted Musculoskeletal/ Metabolic BMN 349 A1ATD (Small Molecule) • Rapidly acting, or
Slide 28: Addressable Patient Population by Product Candidate
Addressable Therapeutic Focus Product Candidate Patient Population 1 BMN 351 DMD (Exon 51 Oligonucleotide) ~8K Musculoskeletal / Metabolic BMN 255 Hyperoxaluria (Small Molecule) ~800K BMN 349 A1ATD (Small Molecule) ~52K BMN 331 HAE (Gene Therapy) ~13K Cardiovascular BMN 293 HCM (Gene Therapy) ~180K
Slide 29: Products Approved in the Last Years
5 Products Approved in the Last 8 Years Enzyme Replacement Therapies Achondroplasia Phenylketonuria Hemophilia A Gene Therapy 29
Slide 30: Sustainable Growth & Profitability
Maximize Base Aggressive Life Indication Revenue Cycle Management Sustainable Growth & Profitability Accelerate Clinical Balance Profits with Pipeline Reinvestments 30
Slide 31: Revenue Growth Enablers and Financial Expectations
Near Term Revenue Growth Enablers Accelerating Revenue Through Mid-Decade & Beyond • Launch VOXZOGO and ROCTAVIAN base indications • Achieve regulatory approval for U.S. $4B - $5B Double-digit ROCTAVIAN Growth $2.09B • Expand VOXZOGO and ROCTAVIAN indications • Continue to grow base portfolio 20221
Slide 32: GAAP to Non-GAAP Reconciliation
GAAP to Non-GAAP Reconciliation P&L Guidance Measures GAAP Net Income P&L Line GAAP Non-GAAP Interest income (expense), net Total Revenue n n Provision for (benefit from) income Gross Profit taxes1 n % of Revenue Depreciation expenses R&D % of n Revenue Amortization expense2 n SG&A % of n Stock-base
Slide 33: Summary of growth strategy
Maximize Base • Durable base business and launch of two potential blockbuster Indication Revenue products drive opportunity to double revenue by mid-decade • VOXZOGO beyond Achondroplasia opportunity significantly out- Aggressive sizes base indication Life Cycle Management • Studies underway to purs
Slide 34: Thank You!
Thank You! “ BiOMARIN NTT
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